BR112022020423A2 - COMPOUNDS AND METHODS FOR SPLICING MODULATION - Google Patents

COMPOUNDS AND METHODS FOR SPLICING MODULATION

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Publication number
BR112022020423A2
BR112022020423A2 BR112022020423A BR112022020423A BR112022020423A2 BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2 BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A BR112022020423 A BR 112022020423A BR 112022020423 A2 BR112022020423 A2 BR 112022020423A2
Authority
BR
Brazil
Prior art keywords
compounds
methods
splicing
splicing modulation
modulation
Prior art date
Application number
BR112022020423A
Other languages
Portuguese (pt)
Inventor
Reynolds Dominic
Leger Serge
W Seiler Michael
A Agrawal Anant
Vaillancourt Frederic
Smith Peter
T Hopper Allen
Prajapati Sudeep
Soueidan Olivier
Original Assignee
Remix Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Remix Therapeutics Inc filed Critical Remix Therapeutics Inc
Publication of BR112022020423A2 publication Critical patent/BR112022020423A2/en

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50Pyridazines; Hydrogenated pyridazines
    • A61K31/502Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with carbocyclic ring systems, e.g. cinnoline, phthalazine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/50Pyridazines; Hydrogenated pyridazines
    • A61K31/5025Pyridazines; Hydrogenated pyridazines ortho- or peri-condensed with heterocyclic ring systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/02Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings
    • C07D401/12Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a chain containing hetero atoms as chain links
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D401/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom
    • C07D401/14Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D403/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/02Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings
    • C07D403/04Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D403/00Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00
    • C07D403/14Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, not provided for by group C07D401/00 containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D413/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms
    • C07D413/14Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and oxygen atoms as the only ring hetero atoms containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D417/00Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/14Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing three or more hetero rings
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D471/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00
    • C07D471/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, at least one ring being a six-membered ring with one nitrogen atom, not provided for by groups C07D451/00 - C07D463/00 in which the condensed system contains two hetero rings
    • C07D471/08Bridged systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04Ortho-condensed systems
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D519/00Heterocyclic compounds containing more than one system of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring system not provided for in groups C07D453/00 or C07D455/00

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  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Health & Medical Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Immunology (AREA)
  • Neurosurgery (AREA)
  • Urology & Nephrology (AREA)
  • Pulmonology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Diabetes (AREA)
  • Biomedical Technology (AREA)
  • Obesity (AREA)
  • Communicable Diseases (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Saccharide Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Replacement Of Web Rolls (AREA)

Abstract

COMPOSTOS E MÉTODOS PARA MODULAÇÃO DE SPLICING. A presente invenção refere-se a compostos e composições relacionadas que, entre outros, modulam o splicing de ácido nucleico, por exemplo, splicing de um pré-mRNA, assim como a métodos de uso dos mesmos.COMPOUNDS AND METHODS FOR SPLICING MODULATION. The present invention relates to compounds and related compositions which, inter alia, modulate nucleic acid splicing, for example, splicing of a pre-mRNA, as well as methods of using the same.

BR112022020423A 2020-04-08 2021-04-08 COMPOUNDS AND METHODS FOR SPLICING MODULATION BR112022020423A2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063007331P 2020-04-08 2020-04-08
US202063044318P 2020-06-25 2020-06-25
US202063072922P 2020-08-31 2020-08-31
US202063126494P 2020-12-16 2020-12-16
PCT/US2021/026481 WO2021207554A1 (en) 2020-04-08 2021-04-08 Compounds and methods for modulating splicing

Publications (1)

Publication Number Publication Date
BR112022020423A2 true BR112022020423A2 (en) 2022-11-29

Family

ID=75690721

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112022020423A BR112022020423A2 (en) 2020-04-08 2021-04-08 COMPOUNDS AND METHODS FOR SPLICING MODULATION

Country Status (14)

Country Link
US (2) US20230159496A1 (en)
EP (1) EP4132646A1 (en)
JP (1) JP2023520916A (en)
KR (1) KR20220167298A (en)
CN (1) CN115551593A (en)
AU (1) AU2021253587A1 (en)
BR (1) BR112022020423A2 (en)
CA (1) CA3174353A1 (en)
CL (1) CL2022002780A1 (en)
CR (1) CR20220566A (en)
IL (1) IL297137A (en)
MX (1) MX2022012678A (en)
TW (1) TW202208344A (en)
WO (2) WO2021207554A1 (en)

Families Citing this family (6)

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Publication number Priority date Publication date Assignee Title
RU2020105929A (en) 2017-08-04 2021-09-06 Скайхоук Терапьютикс, Инк. METHODS AND COMPOSITIONS FOR MODULATING SPLICING
TW202208358A (en) 2020-05-13 2022-03-01 美商Chdi基金會股份有限公司 Htt modulators for treating huntington’s disease
CA3233973A1 (en) * 2021-10-13 2023-04-20 Dominic Reynolds Compounds and methods for modulating nucleic acid splicing
CA3239110A1 (en) * 2021-11-24 2023-06-01 Dominic Reynolds Compounds and methods for modulating splicing
WO2023133225A1 (en) * 2022-01-05 2023-07-13 Remix Therapeutics Inc. Compounds and methods for modulating splicing
US11957759B1 (en) 2022-09-07 2024-04-16 Arvinas Operations, Inc. Rapidly accelerated fibrosarcoma (RAF) degrading compounds and associated methods of use

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DK1163237T3 (en) * 1999-03-17 2004-08-02 Astrazeneca Ab amide derivatives
GB0324790D0 (en) * 2003-10-24 2003-11-26 Astrazeneca Ab Amide derivatives
TWI320783B (en) * 2005-04-14 2010-02-21 Otsuka Pharma Co Ltd Heterocyclic compound
BRPI0817061A2 (en) * 2007-09-12 2015-03-24 Wyeth Llc Pyrrolidinylalkylisoquinolinone and -isoindolinoline derivatives as histamine-3 antagonists
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EP4249472A3 (en) 2015-05-30 2023-12-13 PTC Therapeutics, Inc. Methods for modulating rna splicing
RU2747644C2 (en) * 2015-09-23 2021-05-11 Янссен Фармацевтика Нв Bigeteroaryl-substituted 1,4-benzodiazepines and ways of their use for cancer treatment
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JP6754505B2 (en) * 2017-01-22 2020-09-09 福建広生堂薬業股▲ふん▼有限公司 ASK1 inhibitor, its preparation method and use
JP2020523365A (en) 2017-06-14 2020-08-06 ピーティーシー セラピューティクス,インコーポレーテッド Method of modifying RNA splicing
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WO2020004594A1 (en) 2018-06-27 2020-01-02 株式会社リボルナバイオサイエンス Prophylactic or therapeutic agent for spinal muscular atrophy

Also Published As

Publication number Publication date
MX2022012678A (en) 2023-01-11
KR20220167298A (en) 2022-12-20
EP4132646A1 (en) 2023-02-15
AU2021253587A1 (en) 2022-12-01
WO2021207532A1 (en) 2021-10-14
US20230167093A1 (en) 2023-06-01
WO2021207554A1 (en) 2021-10-14
CL2022002780A1 (en) 2023-04-28
IL297137A (en) 2022-12-01
CN115551593A (en) 2022-12-30
US20230159496A1 (en) 2023-05-25
CA3174353A1 (en) 2021-10-14
JP2023520916A (en) 2023-05-22
CR20220566A (en) 2023-01-23
TW202208344A (en) 2022-03-01

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